SG11201508925WA - Microrna compounds and methods for modulating mir-122 - Google Patents

Microrna compounds and methods for modulating mir-122

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Publication number
SG11201508925WA
SG11201508925WA SG11201508925WA SG11201508925WA SG11201508925WA SG 11201508925W A SG11201508925W A SG 11201508925WA SG 11201508925W A SG11201508925W A SG 11201508925WA SG 11201508925W A SG11201508925W A SG 11201508925WA SG 11201508925W A SG11201508925W A SG 11201508925WA
Authority
SG
Singapore
Prior art keywords
methods
modulating mir
microrna compounds
microrna
compounds
Prior art date
Application number
SG11201508925WA
Other languages
English (en)
Inventor
Balkrishen Bhat
Daniel Hogan
Original Assignee
Regulus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regulus Therapeutics Inc filed Critical Regulus Therapeutics Inc
Publication of SG11201508925WA publication Critical patent/SG11201508925WA/en

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    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4164—1,3-Diazoles
    • A61K31/4178—1,3-Diazoles not condensed 1,3-diazoles and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin
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    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4164—1,3-Diazoles
    • A61K31/4184—1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
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SG11201508925WA 2013-05-01 2014-04-30 Microrna compounds and methods for modulating mir-122 SG11201508925WA (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361818432P 2013-05-01 2013-05-01
US201361822112P 2013-05-10 2013-05-10
US201361839550P 2013-06-26 2013-06-26
US201361895784P 2013-10-25 2013-10-25
US201361898704P 2013-11-01 2013-11-01
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Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ630921A (en) * 2013-05-01 2017-12-22 Regulus Therapeutics Inc Compounds and methods for enhanced cellular uptake
JP2016518842A (ja) 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法
DK3470690T3 (da) * 2013-09-16 2022-01-17 Vaelinge Innovation Ab Monteret produkt
WO2015061536A1 (en) 2013-10-25 2015-04-30 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-21 activity
KR20170083108A (ko) * 2014-11-10 2017-07-17 글락소스미스클라인 인털렉츄얼 프로퍼티 (넘버 2) 리미티드 C형 간염에 대한 장기 작용 약학적 조성물
CA2967184A1 (en) * 2014-11-10 2016-05-19 Glaxosmithkline Intellectual Property (No.2) Limited Combination long acting compositions and methods for hepatitis c
CN109069529B (zh) 2016-03-07 2021-08-20 箭头药业股份有限公司 用于治疗性化合物的靶向配体
WO2017222914A1 (en) 2016-06-21 2017-12-28 Inception 4, Inc. Carbocyclic prolinamide derivatives
PT3472149T (pt) 2016-06-21 2023-11-23 Orion Opthalmology Llc Derivados de prolinamida heterocíclicos
CN116942841A (zh) 2016-09-02 2023-10-27 箭头药业股份有限公司 靶向配体
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
US20210170034A1 (en) * 2018-05-08 2021-06-10 Regulus Therapeutics Inc. Galnac Conjugated Modified Oligonucleotides as MIR-122 Inhibitor Having HCV Antiviral Activity with Reduced Hyperbilirubinemia Side-Effect
TW202019441A (zh) * 2018-07-20 2020-06-01 美商雷格勒斯治療公司 用於經口遞送寡核苷酸之方法
CN112843084A (zh) * 2019-11-27 2021-05-28 中国人民解放军第四军医大学 MiR-122-5p在制备促进创伤愈合药物中的应用
CN114057816A (zh) * 2020-07-30 2022-02-18 浙江柏拉阿图医药科技有限公司 富含腺嘌呤硫代磷酸酯化寡核苷酸及其抗肝炎病毒的应用
CA3233014A1 (en) * 2021-10-08 2023-04-13 Denis Drygin Methods and compositions for avoiding off-target effects
AU2022361062A1 (en) * 2021-10-08 2024-03-21 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease

Family Cites Families (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0759979A4 (en) 1994-05-10 1999-10-20 Gen Hospital Corp ANTISENSE OLIGONUCLEOTIDES INHIBITION OF HEPATITIS C VIRUS
AU1039397A (en) 1995-11-22 1997-06-27 Johns Hopkins University, The Ligands to enhance cellular uptake of biomolecules
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
AU7406700A (en) 1999-10-04 2001-05-10 Exiqon A/S Design of high affinity rnase h recruiting oligonucleotide
JP2003524637A (ja) * 1999-12-23 2003-08-19 エクシコン エ／エス Ｌｎａ修飾オリゴヌクレオチドの治療上の使用
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
AU2002217980A1 (en) 2000-12-01 2002-06-11 Cell Works Inc. Conjugates of glycosylated/galactosylated peptide
CN1344803A (zh) 2001-09-27 2002-04-17 吴昌 核酸靶分子选择及扩增方法
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP2660322A3 (en) 2003-04-17 2013-11-13 Alnylam Pharmaceuticals Inc. Modified iRNA agents
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
ES2382807T3 (es) 2003-08-28 2012-06-13 Takeshi Imanishi Nuevos ácidos nucleicos artificiales del tipo de enlace N-O con reticulación
AU2004275876B2 (en) 2003-09-25 2011-03-31 Coley Pharmaceutical Gmbh Nucleic acid-lipophilic conjugates
KR20070006709A (ko) 2003-12-23 2007-01-11 산타리스 팔마 에이/에스 Ｂｃｌ-2의 조절을 위한 올리고머 화합물
US7626014B2 (en) 2004-04-27 2009-12-01 Alnylam Pharmaceuticals Single-stranded and double-stranded oligonucleotides comprising a 2-arylpropyl moiety
EP2338996A3 (en) 2004-05-04 2013-12-18 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for reducing viral genome amounts in a target cell
WO2006112872A2 (en) 2004-08-04 2006-10-26 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
US20090203132A1 (en) 2004-09-09 2009-08-13 Swayze Eric E Pyrrolidinyl groups for attaching conjugates to oligomeric compounds
EP1838870A2 (en) 2004-12-29 2007-10-03 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF MICRORNAS AND THEIR TARGET MRNAs
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20080206869A1 (en) 2005-01-24 2008-08-28 Avaris Ab Nucleic Acid Complex
AU2006279906B2 (en) 2005-08-10 2012-05-10 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US20090203893A1 (en) 2005-08-29 2009-08-13 Regulus Therapeutics, Llc Antisense compounds having enhanced anti-microrna activity
JP5523705B2 (ja) * 2005-08-29 2014-06-18 レグルス・セラピューティクス・インコーポレイテッド Ｍｉｒ−１２２ａをモジュレートする使用方法
ES2516815T3 (es) 2006-01-27 2014-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en la posición 6
US8129515B2 (en) 2006-01-27 2012-03-06 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
CA3024953A1 (en) 2006-04-03 2007-10-11 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
EA201100813A1 (ru) 2006-04-03 2012-06-29 Сантарис Фарма А/С Фармацевтическая композиция
EP3192788A1 (en) 2006-10-03 2017-07-19 Arbutus Biopharma Corporation Lipid containing formulations
EA018986B1 (ru) 2007-05-01 2013-12-30 Сантарис Фарма А/С Соединения-антагонисты рнк для модуляции активности бета-катенина
AU2008260277C1 (en) 2007-05-30 2014-04-17 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
CN101796062B (zh) 2007-07-05 2014-07-30 Isis制药公司 6-双取代双环核酸类似物
NZ583677A (en) 2007-10-04 2012-06-29 Santaris Pharma As MicroRNAs comprising Locked Nucleic Acid (LNA) units
CN101932709B (zh) 2007-11-26 2014-09-10 桑塔里斯制药公司 靶向雄激素受体的lna拮抗剂
EP3156077B1 (en) 2007-12-04 2022-03-09 Arbutus Biopharma Corporation Targeting lipids
WO2009091972A2 (en) 2008-01-18 2009-07-23 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and small molecules for use in reducing micro rna activity levels and uses thereof
US8361980B2 (en) 2008-03-07 2013-01-29 Santaris Pharma A/S Pharmaceutical compositions for treatment of microRNA related diseases
EP2384197B1 (en) 2008-12-31 2016-04-06 Roche Innovation Center Copenhagen A/S Use of lna apob antisense oligomers for the treatment of acute coronary syndromes
US9034837B2 (en) 2009-04-24 2015-05-19 Roche Innovation Center Copenhagen A/S Pharmaceutical compositions for treatment of HCV patients that are poor-responders to interferon
WO2010144485A1 (en) 2009-06-08 2010-12-16 Miragen Therapeutics CHEMICAL MODIFICATION MOTIFS FOR miRNA INHIBITORS AND MIMETICS
BR112012008865A2 (pt) 2009-10-16 2019-09-24 Glaxo Group Ltd inibidores de hbv anti-sentido.
CA2788600C (en) 2010-02-24 2019-12-03 Arrowhead Research Corporation Compositions for targeted delivery of sirna
JP2013528665A (ja) 2010-03-26 2013-07-11 メルサナ セラピューティックス， インコーポレイテッド ポリヌクレオチドの送達のための修飾ポリマー、その製造方法、およびその使用方法
WO2011130458A2 (en) 2010-04-13 2011-10-20 John Rossi Rna aptamers against baff-r as cell-type specific delivery agents and methods for their use
WO2012007477A1 (en) 2010-07-12 2012-01-19 Santaris Pharma A/S Anti hcv oligomers
AU2011302152B2 (en) * 2010-09-15 2015-06-11 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
CA2816155C (en) 2010-12-17 2020-10-27 Arrowhead Research Corporation Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
ES2605990T3 (es) 2010-12-29 2017-03-17 F. Hoffmann-La Roche Ag Conjugados de molécula pequeña para la administración intracelular de ácidos nucleicos
LT2702155T (lt) * 2011-04-25 2017-05-10 Regulus Therapeutics Inc. Mikro rnr junginiai ir būdai mir-21 aktyvumo moduliavimui
US20140248611A1 (en) * 2011-06-16 2014-09-04 Nippon Steel & Sumikin Eco-Tech Corporation Nucleic acid probe for assaying nucleic acids
US20140127159A1 (en) 2011-06-23 2014-05-08 Stella Aps HCV Combination Therapy
EP2726611A1 (en) 2011-06-30 2014-05-07 Stella ApS Hcv combination therapy
JP2014520772A (ja) 2011-06-30 2014-08-25 ステラ・アンパルトセルスカブ Ｈｃｖ併用療法
WO2013033230A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
WO2013068348A1 (en) 2011-11-07 2013-05-16 Santaris Pharma A/S Lna oligomers for improvement in hepatic function
ES2607681T3 (es) 2011-11-07 2017-04-03 Roche Innovation Center Copenhagen A/S Método de pronóstico para comprobar la eficacia de inhibidores de micro ARN-122 en pacientes VHC+
MX355408B (es) 2012-06-21 2018-04-18 Miragen Therapeutics Inc Inhibidores a base de oligonucleotidos que comprenden un motivo de acido nucleico bloqueado.
EP2900822B1 (en) 2012-09-26 2021-11-24 Guangdong Maijinjia Biotechnology Co., Ltd Oligomers with improved off-target profile
MX363068B (es) 2012-11-15 2019-03-07 Roche Innovation Ct Copenhagen As Conjugados de oligonucleotido.
CA2893801A1 (en) 2013-01-30 2014-08-07 F. Hoffmann-La Roche Ag Lna oligonucleotide carbohydrate conjugates
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
JP2016518842A (ja) 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法
PL2992009T3 (pl) 2013-05-01 2020-11-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji apolipoproteiny(a)
NZ630921A (en) 2013-05-01 2017-12-22 Regulus Therapeutics Inc Compounds and methods for enhanced cellular uptake

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